Research into rare diseases of childhood

doi:10.1001/jama.2013.285873

. 2014 May 7;311(17):1729-30.

doi: 10.1001/jama.2013.285873.

Research into rare diseases of childhood

Mark L Batshaw¹, Stephen C Groft², Jeffrey P Krischer³

Affiliations

¹ Children's National Medical Center, Washington, DC.
² Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland.
³ Pediatric Epidemiology Center, University of South Florida, Tampa.

PMID: 24794360
PMCID: PMC6441966
DOI: 10.1001/jama.2013.285873

Research into rare diseases of childhood

Mark L Batshaw et al. JAMA. 2014.

. 2014 May 7;311(17):1729-30.

doi: 10.1001/jama.2013.285873.

Authors

Mark L Batshaw¹, Stephen C Groft², Jeffrey P Krischer³

Affiliations

¹ Children's National Medical Center, Washington, DC.
² Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland.
³ Pediatric Epidemiology Center, University of South Florida, Tampa.

PMID: 24794360
PMCID: PMC6441966
DOI: 10.1001/jama.2013.285873

No abstract available

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Conflict of interest statement

Conflict of Interest Disclosures: All authors have completed and submitted the ICJME Form for Disclosure of Potential Conflicts of Interest and none were reported.

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References

1. Orphan Drug Act, Pub L No. 97–414, FDA 21, CFR pt 316 (1991).
1. Orphan product designations and approvals. US Food and Drug Administration web site http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. Accessed November 11, 2013.
1. Thorat C, Xu K, Freeman SN, et al. What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis. Pediatrics 2012;129(3):516–521. . - PubMed
1. Research, condition, and disease categories categorical spending project listing by category NIH Rare Diseases web site. http://report.nih.gov/categorical_spending_project_listing.aspx?FY=2012&.... Accessed November 27, 2013.
1. Hanley WB, Linsao LS, Netley C. The efficacy of dietary therapy for phenylketonuria. CMAJ 1971;104(12):1089–1091. . - PMC - PubMed

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[1] Orphan Drug Act, Pub L No. 97–414, FDA 21, CFR pt 316 (1991).

[2] Orphan Drug Act, Pub L No. 97–414, FDA 21, CFR pt 316 (1991).

[3] Orphan product designations and approvals. US Food and Drug Administration web site http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. Accessed November 11, 2013.

[4] Orphan product designations and approvals. US Food and Drug Administration web site http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. Accessed November 11, 2013.

[5] Thorat C, Xu K, Freeman SN, et al. What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis. Pediatrics 2012;129(3):516–521. . - PubMed

[6] Thorat C, Xu K, Freeman SN, et al. What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis. Pediatrics 2012;129(3):516–521. . - PubMed

[7] Research, condition, and disease categories categorical spending project listing by category NIH Rare Diseases web site. http://report.nih.gov/categorical_spending_project_listing.aspx?FY=2012&.... Accessed November 27, 2013.

[8] Research, condition, and disease categories categorical spending project listing by category NIH Rare Diseases web site. http://report.nih.gov/categorical_spending_project_listing.aspx?FY=2012&.... Accessed November 27, 2013.

[9] Hanley WB, Linsao LS, Netley C. The efficacy of dietary therapy for phenylketonuria. CMAJ 1971;104(12):1089–1091. . - PMC - PubMed

[10] Hanley WB, Linsao LS, Netley C. The efficacy of dietary therapy for phenylketonuria. CMAJ 1971;104(12):1089–1091. . - PMC - PubMed

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Research into rare diseases of childhood

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Research into rare diseases of childhood

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